DESCRIPTION:

Four medical centers in CA, CO, MN, and OH are recruiting participants who have specific types of Recessive Dystrophic Epidermolysis Bullosa to test a new, topical treatment. The treatment aims to slow progression of EB wounds through a drug that binds to and stops the cellular messaging system that causes inflammation.

The actual drug and a placebo will be applied 2 to 3x per week to two wound areas, as assigned by the study, for 4 weeks. Home and site visits will follow for regular assessments up to 8 weeks post the last dose.

PATIENT MUST:

• Be 6 years of age or older
• Have at least 1 target wound area
• Have life expectancy > 6 months

THE STUDY INVOLVES:

1. Screening before the treatment.
2. Visit to the study center for assignment of the drug and/or placebo to the targeted wounds.
3. For 4 weeks, the medicines will be applied to the assigned wounds 2 to 3 times per week.
4. Home and site visits will follow for regular assessments up to 8 weeks post the last dose.

LOCATIONS AND CONTACTS:

The study locations include:

• Stanford University School of Medicine, LPCH in Palo Alto, CA
  Contact: Kunju Sridhar  |  650-721-4902  |  [email protected]
• Children’s Hospital Colorado in Aurora, CO
  Contact: Kathleen Peoples  |  720-777-4708 |   [email protected]
• Journey Clinic, Center for Pediatric Blood and Marrow Transplantation in Minneapolis, MN
  Contact: Christen Ebens, MD  |  612-626-8094  |  [email protected]
• Cincinnati Children’s Hospital in Cincinnati, OH
  Contact: Bret Augsburger  |  513-803-9009  |  [email protected]

SPONSOR INFORMATION:

ProQR Therapeutics

Or go online: https://clinicaltrials.gov/ct2/show/NCT03605069